A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)

Completed · Phase 3

Conditions studied: Homozygous Familial Hypercholesterolemia

In brief

The goal of this trial is to study the effects of AEGR-733 on LDL cholesterol, other lipids as well as measures of safety over the long-term.

Key facts

Study ID

NCT00730236

Run by

Aegerion Pharmaceuticals, Inc.

People needed

29

Starts

2007-12-01

Expected to finish

2011-10-01

Last updated by the study team

2018-03-20

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Males and females at least 18 years of age
• Diagnosis of functional homozygous FH by at least one (a-c) of the following clinical criteria:
• documented functional mutation(s) in both LDL receptor alleles or alleles known to affect LDL receptor functionality OR
• skin fibroblast LDL receptor activity less than 20% normal OR
• untreated TC greater than 500 mg/dL AND TG less then 300 mg/dL AND both parents have documented TC greater than 250 mg/dL
• Concurrent lipid lowering medication/apheresis must be stable for at least 6 weeks before the baseline visit and must remain stable for the first 26 weeks.
• Body weight at least 40 kg and less than 136 kg
• Negative screening pregnancy test if female of child-bearing potential (females of child-bearing potential and all males must be following a medically accepted form of contraception)
• Subjects must be willing to comply with all study-related procedures

You may not qualify if…

• Uncontrolled hypertension
• History of chronic renal insufficiency
• History of biopsy proven cirrhosis or abnormal LFTs at screening (AST or ALT greater than 2 x upper limit of normal and/or Total Bilirubin greater than or equal to 1.5 mg/dl unless patient has unconjugated hyperbilirubinemia due to Gilbert's syndrome)
• Chronic hepatitis B or chronic hepatitis C
• Any major surgical procedure occurring less than 3 months prior to the screening visit
• Cardiac insufficiency defined by the NYHA classification as functional Class III or Class IV
• Previous organ transplantation
• History of a non-skin malignancy within the previous 3 years
• Male subjects reporting more than 2 drinks per day or females reporting more than 1 drink per day (1 drink= 12 oz beer, 1 oz hard liquor, 5 oz wine).
• Participation in an investigational drug study within 6 weeks prior to the screening visit
• Known significant gastrointestinal bowel disease or malabsorption such as inflammatory bowel disease or chronic pancreatitis requiring use of daily pancreatic enzymes.
• Serious or unstable medical or psychological conditions that, in the opinion of the investigator, would compromise the subject's safety or successful participation in the study.
• Certain prohibited medications known to be potentially hepatotoxic, especially those that can induce microvesicular or macrovesicular steatosis. These include but are not limited to: accutane, amiodarone, heavy acetaminophen use (4g/day greater than 3 x q week), methotrexate, tetracyclines,and tamoxifen
• Documented diagnosis of any of the following pulmonary conditions: Asthma, Chronic Obstructive Pulmonary Disease (COPD), Idiopathic pulmonary fibrosis
• Documented diagnosis of any of the following liver diseases: Nonalcoholic Steatohepatitis, Alcoholic liver disease, Autoimmune hepatitis, primary biliary cirrhosis, primary sclerosing cholangitis, Wilson's disease, hemochromatosis, alpha 1 anti-trypsin deficiency.
• Current use of corticosteroids or betaine

Where it is running

• Cedars-Sinai Medical Center — Los Angeles, California, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• Robarts Research Institute — London, Ontario, Canada
• Lipid Clinic and University of Montreal Community Genomic Medicine Center — Chicoutimi, Quebec, Canada
• Dipartimento di Medicina Clinica e Della Patalogie Emergenti — Palermo, Sicily, Italy
• Medicina Interna Universitaria — Ferrara, Italy
• Centro Universitario Dislipidemie — Milan, Italy
• Dipartimento di Clinica e Terapia Medica — Roma, Italy
• Cardiology Research — Bloemfontein, South Africa
• University of Capetown — Cape Town, South Africa

Full record on ClinicalTrials.gov

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